Myotonic Dystrophy Type 1Phase 2NCT00577577
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
A Phase 2 study testing rhIGF-I/rhIGFBP-3 in people with Myotonic Dystrophy Type 1, run by Insmed Incorporated at 12 sites in United States.
Trial summary
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients Read more about this study
- Who is it for?
- Ages 21 to 65 years
- Conditions the study is for
- Myotonic Dystrophy Type 1
- What is being tested?
- rhIGF-I/rhIGFBP-3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 12 sitesUnited States
- Study dates
- Dec 2007to 29 Dec 2008
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication
Who cannot join
- Congenital DM1
- Weight greater than 100 kg or body mass index greater than 30 kg/m2
- Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, investigational agent within 60 days of screening
- Current diagnosis or history of malignancy expect for surgically cured skin cancer or pilomatricoma
- Changes in lipid lowering medications during the 3 months prior to screening
- Diaphragmatic weakness such that patients are unable to tolerate the supine position, or swallowing impairment such that patients are unable to maintain nutrition without use of gastrostomy.
- Major psychiatric illness (major depression, bipolar disorder or schizophrenia) within twelve months of screening
- History of non-compliance with other therapies
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Ohio State University Medical Center
Columbus, Ohio, 43210
Universit of Texas Medical Branch
Galveston, Texas, 77555-0539
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Minnesota
Minneapolis, Minnesota, 55455
University of California Irvine Medical Center; MDA, ALS and Neuromuscular Center
Orange, California, 92868
Oregan Health and Science University
Portland, Oregon, 97239
University of Rochester, Neuromuscular Disease Center
Rochester, New York, 14642
University of California, Davis
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84112
University of Texas Health Science Center
San Antonio, Texas, 78229
Washington University Medical School
St Louis, Missouri, 63110
About this study
Official title: A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients
Read the detailed description
Efficacy Measures:
Endurance, Ambulation, Cognitive function, Insulin resistance, Cholesterol and triglycerides, Muscle function and strength, Pain, Gastrointestinal function, Quality of life
MINIMUM INCLUSION CRITERIA
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Age 21 to 65 years (inclusive)
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication
- Conditions
- Myotonic Dystrophy Type 1
- Being tested
- rhIGF-I/rhIGFBP-3, placebo
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhIGF-I/rhIGFBP-3Drug
1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
placeboDrug
1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
| Study group | Role | What they receive |
|---|---|---|
| rhIGF-I/rhIGFBP-3 | Experimental | Not described Receives: Drug: rhIGF-I/rhIGFBP-3 |
| Placebo | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance
The 6MWT measured the distance in meters that participants were able to walk over a total of six minutes. After a 10 minute resting period, the participants completed the 6MWT on a hard, flat surface at baseline and at Week 24.
Measured: Baseline and Week 24
Change From Baseline in Daily Step Count
The number of steps taken per day was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a negative change from baseline indicates a decrease in the number of daily steps.
Measured: Baseline and Week 24
Peak Activity Index: Change From Baseline in Number of Steps Walked Per Minute During the 30 Minute Period of Fastest Walking
The peak activity index measures the number of steps walked in the 30 minutes of fastest walking that occurred in a 24 hour period. This was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of steps walked during the 30 minute period of fastest walking.
Measured: Baseline and Week 24
Sustained Activity Index: Change From Baseline in the Highest Number of Steps Walked Per Minute Over 20 Minutes of Activity
The sustained activity index measures the highest number of steps sustained over a continuous 20 minute period. This was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of steps walked over 20 minutes of activity.
Measured: Baseline and Week 24
Change From Baseline in the Percentage of Time That Participants Spent Inactive
Change from baseline scores were measured where a negative change from baseline indicates less time spent inactive.
Measured: Baseline and Week 24
Change From Baseline in Time Taken for Participants to Ascend and Descend 4 Stairs
Participants were timed on their ability to climb up 4 stairs and timed separately to climb down 4 stairs at baseline and at week 24. The stairs were free-standing or the same flight of stairs was used at each assessment. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the time taken for paticipants to ascend or descend 4 stairs.
Measured: Baseline and Week 24
Change From Baseline in Time Taken to Traverse 30 Feet
Participants were timed on their ability to travel 30 feet on the same surface at each assessment at baseline and at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the time taken to travel 30 feet.
Measured: Baseline and Week 24
Change From Baseline in Purdue Pegboard Test Scores
The Purdue Pegboard Test consists of a board with two sets of 25 holes, 4 concave cups, and a number of small metal pins. Participants were required to pick up the pins from a holder and place them in the holes as quickly as possible over 30 seconds with their dominant hand. The score was calculated as the number of pins placed into holes in 30 seconds and was measured at baseline and Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of pins placed in the board.
Measured: Baseline and Week 24
Change From Baseline in Forced Vital Capacity (FVC) Volume While Sitting or Lying Down
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible, as measured by spirometry.
Measured: Baseline and Week 24
Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted While Sitting or Lying Down
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible, as measured by spirometry.
Measured: Baseline and Week 24
Change From Baseline in Manual Muscle Test (MMT) Scores
The MMT was used to assess muscle strength in the distal muscles and the proximal muscles. Distal muscles assessments included wrist extension, wrist flexion, ankle dorsiflexion and plantarflexion. Proximal muscle assessments included shoulder abduction, elbow extension, elbow flexion, hip extension, hip abduction, hip flexion, knee extension and knee flexion.
In MMT, each muscle assessment was given a score of 0 to 5, where 0 indicated 'no contraction palpable' and 5 indicated 'normal strength'. The scores from each muscle were summed and the maximum overall score of all measured muscles was 140, the maximum distal score was 40 and the maximum proximal score was 80. Higher scores indicated higher muscle strength. Change from baseline scores were measured where a positive change from baseline indicates an improvement in muscle strength.
Measured: Baseline and Week 24
Change From Baseline in Selective Reminding Test T-Scores (Total Word and Delayed Words)
The Selective Reminding Test measures verbal learning and memory and involves remembering a verbal list of 12 words. Participants were required to recall the 12 words presented. Words that were missed on recall were presented again, and the process was repeated until all 12 words were correctly recalled. The total word list recall and delayed recall were calculated as T-scores. Raw scores were converted to T-score using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Measured: Baseline and Week 24
Change From Baseline in Selective Reminding Test Raw Scores (Cued Recall and Recognition)
The Selective Reminding Test measures verbal learning and memory and involves remembering a verbal list of 12 words. Participants were required to recall the 12 words presented. Words that were missed on recall were presented again, and the process is repeated until all 12 words were correctly recalled. The cued recall scores ranged from 0-11 and multiple choice recognition scores ranged from 0-12. Change from baseline scores were measured where a positive change from baseline indicates an improvement in verbal recall and memory.
Measured: Baseline and Week 24
Change From Baseline in Rey Complex Figure (RCF) Test Scores
The Rey Complex Figure Test (RCFT) assesses visuospatial construction ability and visual memory through four different tests: copy (copying a complex geometric figure), immediate recall of the figure (drawing figure from memory at 3 minutes), delayed recall (drawing figure at 30 minutes after initial copy), and recognition score (selecting individual parts of the figure from sketches provided). Copy performances are divided into 18 components with a maximum score of 2 each. The maximum score for each figure is 36.
Measured: Baseline and Week 24
Change From Baseline in Letter-Number Sequencing (LNS) Test Scores
The LNS test from the Welchsler Adult Intelligence Scale-III was used to assess working memory. The test required that participants recall, in order, numbers and letters presented in an unordered sequence. The number of items is 21. With each item being marked 0 if reported incorrectly or 1 if reported correctly, the maximum score is 21. Raw scores were converted into T-scores using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Measured: Baseline and Week 24
Change From Baseline in Trail Making Test (TMT) Scores
The TMT assesses executive function, sequencing, mental flexibility, visual spanning speed and motor function. In TMT Part A, the participant had to draw lines in the correct order between 25 numbers randomly arranged on the page. In TMT Part B, the participant had to draw lines between 25 numbers and letters in alternating order (e.g., 1-A-2-B...etc). Times for Part A and Part B were used to derive T-scores which can range from a minimum of 0 and a maximum of 100. Raw scores were converted into T-scores using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Measured: Baseline and Week 24
Change From Baseline in the Stroop Color Word Test Scores
The Stroop Color Word Test measures selective attention and cognitive flexibility. The test has three parts, the Word test (reading words), the Color test (naming the ink color in which words are displayed) and the Color-Word test (saying the ink color not reading the word). An interference score was calculated from the Color, Word and Color-Word scores and is an indication of how well a person can complete a task while disregarding interfering information. Raw scores were converted into T-scores using available normative data. Scores range from 0 to 100. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Measured: Baseline and Week 24
Change From Baseline to Week 24 Scores on the Beck Depression Inventory II (BDI-II) Questionnaire
BDI-II is a validated self-reported instrument of 21 questions which are each scored 0-3. Total scores range from 0-63, with higher score totals indicating more severe depression symptoms. {0-9: indicates minimal depression; 0-18: indicates mild depression; 19-29: indicates moderate depression; 30-63: indicates severe depression. Lower scores indicate no or minimal depression, with a maximum total score of 63.
Measured: Baseline and Week 24
Change From Baseline in Average Fasting Glucose Concentration in the Blood
Participants were administered 75 grams (g) glucose solution prior to administration of the first dose of IPlex™ and after administration of the last dose. A 2-hour oral glucose tolerance test (OGTT) was performed under fasted conditions.
Measured: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
Change From Baseline in Average Fasting Insulin Concentration in the Blood
Participants were administered 75 g glucose solution prior to administration of the first dose of IPLEX™ and after administration of the last dose. A 2-hour OGTT was performed under fasted conditions.
Measured: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
Change From Baseline in Qualitative Insulin Sensitivity Check Index (QUICKI)
The QUICKI is based on fasting glucose and insulin measurements and are calculated using the following equation:
QUICKI = 1/[ log(fasting glucose in mg/dL) + log (fasting insulin in uU/mL) ]
Measured: Baseline and Week 24
Change From Baseline in Insulin Sensitivity Index-Matsuda (ISI-Matsuda)
The ISI-Matsuda is based on the average glucose and insulin values obtained during the entire oral glucose tolerance test and are calculated using the following equation:
ISI = 10,000 / √ [ fasting glucose (mg/dL) x fasting insulin(uU/mL) x mean glucose x mean insulin ]
Measured: Baseline and Week 24
Change From Baseline in Total Blood Cholesterol Level
A negative change from baseline indicates a decrease in total blood cholesterol level.
Measured: Baseline and Week 24
Change From Baseline in Total Blood Low-density Lipoproteins (LDL) Level
A positive change from baseline indicates an increase in total blood LDL level.
Measured: Baseline and Week 24
Change From Baseline in Total Blood High-density Lipoproteins (HDL) Level
A negative change from baseline indicates a decrease in total blood HDL level.
Measured: Baseline and Week 24
Change From Baseline in Total Blood Triglycerides Level
A negative change from baseline indicates a decrease in total blood triglycerides level.
Measured: Baseline and Week 24
Change From Baseline in Gastro-esophageal Reflux Disease (GERD) Symptom Frequency Questionnaire (GSFQ) Scores
The GSFQ contains 6 questions that assess the frequency of certain GERD symptoms and their impact on daily life. Scores were converted and reported out of 100 with higher scores indicative of more frequent and intense GERD symptoms. Change from baseline scores were measured where a negative change from baseline indicates less frequent and intense GERD symptoms.
Measured: Baseline and Week 24
Change From Baseline in Gastrointestinal Symptom Rating Scale for Irritable Bowel Syndrome (GSRS-IBS) Questionnaire Scores
The GSRS-IBS has 13 questions aimed at identifying the frequency and intensity of IBS symptoms during the past week. Answers are given a score from 1 (no discomfort at all) to 7 (very severe discomfort). A total score was calculated and ranged from 0 to 78. A lower score indicates less discomfort from IBS symptoms. Change from baseline scores were measured where a positive change from baseline indicates increased discomfort from IBS symptoms.
Measured: Baseline and Week 24
Change From Baseline in Swallowing Disturbance Questionnaire (SDQ) Scores
The SDQ had 15 questions relating to the oral phase and pharyngeal phase of swallowing. Answers for 14 questions were assigned a number (0-3) based on a 4-point verbal scale (never, seldom, frequently, very frequently) and the last question was a yes or no question about respiratory infections. A higher score is indicative of greater swallowing issues with 44.5 as the highest possible score. A total score of ≥ 11 suggests impairment. Change from baseline scores were measured where a positive change from baseline indicates increased swallowing impairment.
Measured: Baseline and Week 24
Change From Baseline in Short Form (36) (SF-36) Questionnaire Scores
The SF-36 is a 36-item questionnaire that evaluates quality of life through physical and mental health across eight scales, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability. Change from baseline scores were measured where a positive change from baseline indicates an improvement in quality of life.
Measured: Baseline and Week 24
Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Severity Scores
The BPI contains 15 questions that assess the severity of pain and its impact or interference on functions of daily life. Pain severity was measured as the mean of 7 items of the questionnaire on an 11-point scale where 0 indicates no pain and 10 indicates the worst pain. A higher score indicates greater pain. Categories assessed include worst pain in 24 hours and average pain. Change from baseline scores were measured where a positive change from baseline indicates a worsening in pain.
Measured: Baseline and Week 24
Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Interference Scores
The BPI contains 15 questions that assess the severity of pain and its impact or interference on functions of daily life. Pain interference is measured as the mean of 7 items on an 11-point scale where 0 indicates no interference and 10 indicates complete interference. A higher score indicates greater impairment due to pain. Change from baseline scores were measured where a positive change from baseline indicates a worsening of interference due to pain.
Measured: Baseline and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2007 (estimated)
- Study startDec 2007
- Primary completion29 Dec 2008
- Study completion29 Dec 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myotonic Dystrophy Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Muscular Dystrophy Association
- Responsible party
- Sponsor
- Investigators
- Richard Moxley, M.D., Study chair, University of Rochester Neuromuscular Disease Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00577577
- Sponsor's protocol ID
- INSM-110-1001
- Status last verified
- Dec 2021
- First submitted
- 18 Dec 2007
- Last updated
- 6 Jan 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT00577577
- Acronym / sponsor ID
- INSM-110-1001
- Conditions
- Myotonic Dystrophy Type 1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Muscular Dystrophy Association
- Study start
- Dec 2007 (actual)
- Primary completion
- 29 Dec 2008 (actual)
- Study completion
- 29 Dec 2008 (actual)
- First posted
- 20 Dec 2007 (estimated)
- Results
- Posted 6 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients
Detailed description
Efficacy Measures:
Endurance, Ambulation, Cognitive function, Insulin resistance, Cholesterol and triglycerides, Muscle function and strength, Pain, Gastrointestinal function, Quality of life
MINIMUM INCLUSION CRITERIA
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Age 21 to 65 years (inclusive)
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rhIGF-I/rhIGFBP-3 | Experimental | Drug: rhIGF-I/rhIGFBP-3 |
| Placebo | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 21 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication
Key exclusion criteria (8 listed)
- Congenital DM1
- Weight greater than 100 kg or body mass index greater than 30 kg/m2
- Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, investigational agent within 60 days of screening
- Current diagnosis or history of malignancy expect for surgically cured skin cancer or pilomatricoma
- Changes in lipid lowering medications during the 3 months prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rhIGF-I/rhIGFBP-31.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit. | Drug | rhIGF-I/rhIGFBP-3 |
| placebo1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit. | Drug | Placebo |
Primary outcome
Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance
Time frame: Baseline and Week 24
The 6MWT measured the distance in meters that participants were able to walk over a total of six minutes. After a 10 minute resting period, the participants completed the 6MWT on a hard, flat surface at baseline and at Week 24.
Change From Baseline in Daily Step Count
Time frame: Baseline and Week 24
The number of steps taken per day was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a negative change from baseline indicates a decrease in the number of daily steps.
Peak Activity Index: Change From Baseline in Number of Steps Walked Per Minute During the 30 Minute Period of Fastest Walking
Time frame: Baseline and Week 24
The peak activity index measures the number of steps walked in the 30 minutes of fastest walking that occurred in a 24 hour period. This was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of steps walked during the 30 minute period of fastest walking.
Sustained Activity Index: Change From Baseline in the Highest Number of Steps Walked Per Minute Over 20 Minutes of Activity
Time frame: Baseline and Week 24
The sustained activity index measures the highest number of steps sustained over a continuous 20 minute period. This was measured using a step activity monitor for 7 days at baseline and again at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of steps walked over 20 minutes of activity.
Change From Baseline in the Percentage of Time That Participants Spent Inactive
Time frame: Baseline and Week 24
Change from baseline scores were measured where a negative change from baseline indicates less time spent inactive.
Change From Baseline in Time Taken for Participants to Ascend and Descend 4 Stairs
Time frame: Baseline and Week 24
Participants were timed on their ability to climb up 4 stairs and timed separately to climb down 4 stairs at baseline and at week 24. The stairs were free-standing or the same flight of stairs was used at each assessment. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the time taken for paticipants to ascend or descend 4 stairs.
Change From Baseline in Time Taken to Traverse 30 Feet
Time frame: Baseline and Week 24
Participants were timed on their ability to travel 30 feet on the same surface at each assessment at baseline and at Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the time taken to travel 30 feet.
Change From Baseline in Purdue Pegboard Test Scores
Time frame: Baseline and Week 24
The Purdue Pegboard Test consists of a board with two sets of 25 holes, 4 concave cups, and a number of small metal pins. Participants were required to pick up the pins from a holder and place them in the holes as quickly as possible over 30 seconds with their dominant hand. The score was calculated as the number of pins placed into holes in 30 seconds and was measured at baseline and Week 24. Change from baseline scores were measured where a positive change from baseline indicates an improvement in the number of pins placed in the board.
Change From Baseline in Forced Vital Capacity (FVC) Volume While Sitting or Lying Down
Time frame: Baseline and Week 24
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible, as measured by spirometry.
Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted While Sitting or Lying Down
Time frame: Baseline and Week 24
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible, as measured by spirometry.
Change From Baseline in Manual Muscle Test (MMT) Scores
Time frame: Baseline and Week 24
The MMT was used to assess muscle strength in the distal muscles and the proximal muscles. Distal muscles assessments included wrist extension, wrist flexion, ankle dorsiflexion and plantarflexion. Proximal muscle assessments included shoulder abduction, elbow extension, elbow flexion, hip extension, hip abduction, hip flexion, knee extension and knee flexion. In MMT, each muscle assessment was given a score of 0 to 5, where 0 indicated 'no contraction palpable' and 5 indicated 'normal strength'. The scores from each muscle were summed and the maximum overall score of all measured muscles was 140, the maximum distal score was 40 and the maximum proximal score was 80. Higher scores indicated higher muscle strength. Change from baseline scores were measured where a positive change from baseline indicates an improvement in muscle strength.
Change From Baseline in Selective Reminding Test T-Scores (Total Word and Delayed Words)
Time frame: Baseline and Week 24
The Selective Reminding Test measures verbal learning and memory and involves remembering a verbal list of 12 words. Participants were required to recall the 12 words presented. Words that were missed on recall were presented again, and the process was repeated until all 12 words were correctly recalled. The total word list recall and delayed recall were calculated as T-scores. Raw scores were converted to T-score using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Change From Baseline in Selective Reminding Test Raw Scores (Cued Recall and Recognition)
Time frame: Baseline and Week 24
The Selective Reminding Test measures verbal learning and memory and involves remembering a verbal list of 12 words. Participants were required to recall the 12 words presented. Words that were missed on recall were presented again, and the process is repeated until all 12 words were correctly recalled. The cued recall scores ranged from 0-11 and multiple choice recognition scores ranged from 0-12. Change from baseline scores were measured where a positive change from baseline indicates an improvement in verbal recall and memory.
Change From Baseline in Rey Complex Figure (RCF) Test Scores
Time frame: Baseline and Week 24
The Rey Complex Figure Test (RCFT) assesses visuospatial construction ability and visual memory through four different tests: copy (copying a complex geometric figure), immediate recall of the figure (drawing figure from memory at 3 minutes), delayed recall (drawing figure at 30 minutes after initial copy), and recognition score (selecting individual parts of the figure from sketches provided). Copy performances are divided into 18 components with a maximum score of 2 each. The maximum score for each figure is 36.
Change From Baseline in Letter-Number Sequencing (LNS) Test Scores
Time frame: Baseline and Week 24
The LNS test from the Welchsler Adult Intelligence Scale-III was used to assess working memory. The test required that participants recall, in order, numbers and letters presented in an unordered sequence. The number of items is 21. With each item being marked 0 if reported incorrectly or 1 if reported correctly, the maximum score is 21. Raw scores were converted into T-scores using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Change From Baseline in Trail Making Test (TMT) Scores
Time frame: Baseline and Week 24
The TMT assesses executive function, sequencing, mental flexibility, visual spanning speed and motor function. In TMT Part A, the participant had to draw lines in the correct order between 25 numbers randomly arranged on the page. In TMT Part B, the participant had to draw lines between 25 numbers and letters in alternating order (e.g., 1-A-2-B...etc). Times for Part A and Part B were used to derive T-scores which can range from a minimum of 0 and a maximum of 100. Raw scores were converted into T-scores using available normative data. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Change From Baseline in the Stroop Color Word Test Scores
Time frame: Baseline and Week 24
The Stroop Color Word Test measures selective attention and cognitive flexibility. The test has three parts, the Word test (reading words), the Color test (naming the ink color in which words are displayed) and the Color-Word test (saying the ink color not reading the word). An interference score was calculated from the Color, Word and Color-Word scores and is an indication of how well a person can complete a task while disregarding interfering information. Raw scores were converted into T-scores using available normative data. Scores range from 0 to 100. Change from baseline T-scores were measured where a positive change from baseline indicates improvement in performance.
Change From Baseline to Week 24 Scores on the Beck Depression Inventory II (BDI-II) Questionnaire
Time frame: Baseline and Week 24
BDI-II is a validated self-reported instrument of 21 questions which are each scored 0-3. Total scores range from 0-63, with higher score totals indicating more severe depression symptoms. {0-9: indicates minimal depression; 0-18: indicates mild depression; 19-29: indicates moderate depression; 30-63: indicates severe depression. Lower scores indicate no or minimal depression, with a maximum total score of 63.
Change From Baseline in Average Fasting Glucose Concentration in the Blood
Time frame: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
Participants were administered 75 grams (g) glucose solution prior to administration of the first dose of IPlex™ and after administration of the last dose. A 2-hour oral glucose tolerance test (OGTT) was performed under fasted conditions.
Change From Baseline in Average Fasting Insulin Concentration in the Blood
Time frame: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
Participants were administered 75 g glucose solution prior to administration of the first dose of IPLEX™ and after administration of the last dose. A 2-hour OGTT was performed under fasted conditions.
Change From Baseline in Qualitative Insulin Sensitivity Check Index (QUICKI)
Time frame: Baseline and Week 24
The QUICKI is based on fasting glucose and insulin measurements and are calculated using the following equation: QUICKI = 1/[ log(fasting glucose in mg/dL) + log (fasting insulin in uU/mL) ]
Change From Baseline in Insulin Sensitivity Index-Matsuda (ISI-Matsuda)
Time frame: Baseline and Week 24
The ISI-Matsuda is based on the average glucose and insulin values obtained during the entire oral glucose tolerance test and are calculated using the following equation: ISI = 10,000 / √ [ fasting glucose (mg/dL) x fasting insulin(uU/mL) x mean glucose x mean insulin ]
Change From Baseline in Total Blood Cholesterol Level
Time frame: Baseline and Week 24
A negative change from baseline indicates a decrease in total blood cholesterol level.
Change From Baseline in Total Blood Low-density Lipoproteins (LDL) Level
Time frame: Baseline and Week 24
A positive change from baseline indicates an increase in total blood LDL level.
Change From Baseline in Total Blood High-density Lipoproteins (HDL) Level
Time frame: Baseline and Week 24
A negative change from baseline indicates a decrease in total blood HDL level.
Change From Baseline in Total Blood Triglycerides Level
Time frame: Baseline and Week 24
A negative change from baseline indicates a decrease in total blood triglycerides level.
Change From Baseline in Gastro-esophageal Reflux Disease (GERD) Symptom Frequency Questionnaire (GSFQ) Scores
Time frame: Baseline and Week 24
The GSFQ contains 6 questions that assess the frequency of certain GERD symptoms and their impact on daily life. Scores were converted and reported out of 100 with higher scores indicative of more frequent and intense GERD symptoms. Change from baseline scores were measured where a negative change from baseline indicates less frequent and intense GERD symptoms.
Change From Baseline in Gastrointestinal Symptom Rating Scale for Irritable Bowel Syndrome (GSRS-IBS) Questionnaire Scores
Time frame: Baseline and Week 24
The GSRS-IBS has 13 questions aimed at identifying the frequency and intensity of IBS symptoms during the past week. Answers are given a score from 1 (no discomfort at all) to 7 (very severe discomfort). A total score was calculated and ranged from 0 to 78. A lower score indicates less discomfort from IBS symptoms. Change from baseline scores were measured where a positive change from baseline indicates increased discomfort from IBS symptoms.
Change From Baseline in Swallowing Disturbance Questionnaire (SDQ) Scores
Time frame: Baseline and Week 24
The SDQ had 15 questions relating to the oral phase and pharyngeal phase of swallowing. Answers for 14 questions were assigned a number (0-3) based on a 4-point verbal scale (never, seldom, frequently, very frequently) and the last question was a yes or no question about respiratory infections. A higher score is indicative of greater swallowing issues with 44.5 as the highest possible score. A total score of ≥ 11 suggests impairment. Change from baseline scores were measured where a positive change from baseline indicates increased swallowing impairment.
Change From Baseline in Short Form (36) (SF-36) Questionnaire Scores
Time frame: Baseline and Week 24
The SF-36 is a 36-item questionnaire that evaluates quality of life through physical and mental health across eight scales, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability. Change from baseline scores were measured where a positive change from baseline indicates an improvement in quality of life.
Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Severity Scores
Time frame: Baseline and Week 24
The BPI contains 15 questions that assess the severity of pain and its impact or interference on functions of daily life. Pain severity was measured as the mean of 7 items of the questionnaire on an 11-point scale where 0 indicates no pain and 10 indicates the worst pain. A higher score indicates greater pain. Categories assessed include worst pain in 24 hours and average pain. Change from baseline scores were measured where a positive change from baseline indicates a worsening in pain.
Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Interference Scores
Time frame: Baseline and Week 24
The BPI contains 15 questions that assess the severity of pain and its impact or interference on functions of daily life. Pain interference is measured as the mean of 7 items on an 11-point scale where 0 indicates no interference and 10 indicates complete interference. A higher score indicates greater impairment due to pain. Change from baseline scores were measured where a positive change from baseline indicates a worsening of interference due to pain.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Moxley, M.D. study chair
University of Rochester Neuromuscular Disease Center
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhIGF-I/rhIGFBP-3 in people with Myotonic Dystrophy Type 1, run by Insmed Incorporated at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jan 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance (time frame: Baseline and Week 24). The study lists 32 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2022.